Eteplirsen approved for treating Duchenne Muscular Dystrophy

CHICAGO — The Muscular Dystrophy Association today celebrated news of the U.S. Food and Drug Administration’s decision to grant accelerated approval for eteplirsen, the first disease-modifying drug to treat the most common childhood form of muscular dystrophy.

Accelerated approval of the drug, which will treat a subset of those living with Duchenne muscular dystrophy, is an important step forward in the development of therapies for neuromuscular diseases and marks an historic achievement for the entire DMD community.

“Today has been a long time in the making,” said MDA President and CEO Steven M. Derks. “This is the outcome MDA dreamed of 25 years ago when it was the first to invest in the breakthrough research that led to development of eteplirsen. Throughout this process we have seen the undeniable strength of our community to rally behind MDA’s commitment to find treatments for our families. This is an important victory, and we are honored to stand shoulder-to-shoulder with everyone who has fought to make this day a reality.”

Serepta Therapeutics

Approval to market eteplirsen was given to pharmaceutical company Sarepta Therapeutics. Eteplirsen will be the first disease-modifying drug on the market in the United States to treat DMD, and approximately 13 percent of DMD patients potentially may be eligible for treatment. Under the terms of the FDA’s accelerated approval, Sarepta must conduct a clinical trial of eteplirsen to confirm clinical benefit.

The news comes following an historic turnout at the FDA’s advisory committee hearing in April, which brought a record-breaking number of families, members of the medical community and supporters to Washington to testify on behalf of the DMD community in favor of treatment options for Duchenne.

MDA Executive Vice President and Chief Medical and Scientific Officer Valerie A. Cwik, M.D., provided compelling oral testimony at the hearing, in addition to other MDA appeals to the FDA, urging the agency to consider the totality of the data and utilize maximum regulatory flexibility in its review of the drug.

“For our families, therapy options can’t come soon enough,” Cwik said. “MDA is eager for this treatment to get into the hands of those whom it can help, forever grateful to our partners, supporters, and, most importantly, our families, who all helped turn hope into a treatment that can change the course of Duchenne. We fully expect this accelerated approval will be an inspiration and a catalyst to more innovation and follow-on funding for drug development across the board.”

MDA has invested more than $200 million in DMD research and has been central to the development of the exon skipping approach from the beginning in the 1990s, having funded foundational work upon which the strategy was built as well as extensive research into the strategy since that time. MDA supported the early development of eteplirsen via funding to Steve Wilton, then at the University of Western Australia in Perth, who pioneered the exon skipping technique that allows eteplirsen to work.

This year, MDA already has committed more than $17 million to research, as it takes a unique big-picture perspective across the spectrum of muscle-debilitating diseases that take away everyday abilities such as walking, talking and hugging. With the help of its supporters, MDA plans to double its research spend targeting treatments and clinical trials by the year 2020.-USNewswire